On 26 October at the 7th Joint ECTRIMS-ACTRIMS conference held in Paris, a panel of neurologists discussed the idea of using real-world evidence (RWE ) to design personalised treatments for multiple sclerosis (MS ). The use of personalised medicine based on RWE is likely to produce better treatment outcomes for patients.
Traditionally, the treatment algorithm for MS is stratified. Doctors often will use drugs on patients based on their own experience and clinical guidelines; they often start with first-line, safe drugs such as interferons and Copaxone to see whether patients respond to them, and switch to higher-efficacy drugs if patients fail to respond. This approach is crude and requires a certain degree of trial and error, which often misses the best treatment window for the patient, and early treatment is key to slow disease progression in MS.
Personalised medicine based on RWE adopts a data-driven approach and aims to design the best treatment option tailored to individual patients, using predictions from a large data set. Unlike in the tightly controlled environment in clinical trials, where a limited number of patients are carefully selected and many aspects of treatments are closely monitored, in the real world, there is a much larger patient population with varying traits and different complications that might be overlooked or excluded in clinical trials. The panel believes a big data RWE approach will produce better treatment outcomes than the traditional stratified approach, as every patient is different.
However, as the panel pointed out, this is not an easy task. One of the main challenges is ensuring good data quality. For example, in clinical trials, treatments are often randomized and blinded to the patients and physicians; however, in the real world, both the patients and physicians know which drug was used, and this may lead to bias. Additionally, there are other challenges in merging different MS databases across different countries and geographies, as there are inconsistencies between guidelines, clinical practices, and how the data were recorded.
Nevertheless, the big data RWE approach is an interesting idea and regulatory agencies, in particular the EMA, are considering adopting RWE to replace Phase IV trials. With more data available and better awareness of RWE, we may eventually move away from the traditional trial-and-error approach and towards personalised medicine, which will benefit MS patients in the long run.
GlobalData (2017). PharmaPoint: Multiple Sclerosis – Global Drug Forecast and Market Analysis to 2026, to be published